Evidence map›Paper›PMID 39026931›Full record

ArticleMedical research archives2024

Considerations on Design and Analysis of External Control in Pediatric Oncology.

Jingjing Ye, Haitao Pan, Gregory Reaman, Satrajit Roychoudhury, Chengxing Lu, Lindsay A Renfro, Yuan Ji, Rong Liu, Ying Yuan, Weidong Zhang

Abstract read
In one paragraph

Article in Medical research archives, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

10 authors.

Jingjing YeGlobal Statistics and Data Sciences (GSDS), BeiGene USA, Fulton, MD.
Haitao PanDepartment of Biostatistics, St Jude Children's Research Hospital, Memphis, TN.
Gregory ReamanNational Cancer Institute (NCI), National Institute of Health (NIH).
Satrajit RoychoudhuryStatistical Research and Innovation, Pfizer Inc.
Chengxing LuOncology Biometrics, AstraZeneca, Waltham, Massachusetts.
Lindsay A RenfroDivision of Biostatistics, University of Southern California and Children's Oncology Group, Los Angeles, CA.
Yuan JiUniversity of Chicago, Chicago, IN.
Rong LiuBiostatistics and Data Management, Regeneron Pharmaceuticals, Tarrytown NY 10591.
Ying YuanDepartment of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX.
Weidong ZhangSana Biotechnology, Cambridge, Massachusetts.

Funding

NCTN BIQSFP ANBL1531 (NRT)U10CA180886 · NCI · PUBLIC HEALTH INSTITUTE · PI Douglas S. Hawkins · 2014 to 2026
$390.6M
COG SDMC - Statistics CoreU10CA180899 · NCI · UNIVERSITY OF SOUTHERN CALIFORNIA · PI TODD A ALONZO · 2014 to 2026
$132.8M
NCI NIH HHS U10 CA180886NCI NIH HHS U10 CA180899
6 · The paper itself

Abstract

Pediatric cancer consists of a diverse group of rare diseases. Due to limited patient populations, standard randomized and controlled trials are often infeasible. As a result, single-arm trials are common in pediatric oncology and the use of external controls is often desirable or necessary to help generate actionable evidence and contextualize trial results. In this paper, we illustrate unique features in pediatric oncology clinical trials and describe their impact on the use of external controls. Various types of relevant external control data sources are described in terms of their utility and drawbacks. Statistical methodologies and design implications with external control are discussed. Two recent case studies using external controls to support pediatric oncology drug development are described in detail.

Indexed as

Bayesian analysiscombination therapyexternal controlsPediatric oncologyprior clinical trialregistry

Identifiers

PMID39026931
PMCPMC11257159

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.